Gene Therapy: Investment Hotspot or Ageing Novelty?

Researchers have long dreamed of treating or even preventing genetic diseases—such as Alzheimer’s, cystic fibrosis, Parkinson’s and inherited blindness—by manipulating our genomes. However, just over 50 years on from the first recorded success of heritable gene transfer into a human cell line [1], there is still little evidence of the predicted boon to medicine. So we can’t help but ask: is there actually any light at the end of the tunnel for gene therapy?

In November 2012, the European Medicine Agency approved their first ever gene therapy product, Glybera, developed by the Dutch biotech company uniQure to treat lipoprotein lipase deficiency; uniQure have also just filed for a $75 million IPO, as announced on 2nd January 2014. Furthermore, the emergence of designer nuclease technologies—ZFN and TALEN [2], and most recently CRISPR [3]—appeared to have breathed new life into a field that barely scraped into the new millennium [4].

The growth trajectory of the gene therapy field was cut short 15 years ago by the death of Jesse Gelsinger (Figure 1). Jesse, an 18-year-old lifetime sufferer of a mild enzyme deficiency, undertook a phase I clinical trial at the University of Pennsylvania in 1999 to help test the safety of an adenovirus-derived treatment to replace the gene for the enzyme he lacked; he died of multiple organ failure four days after his first dose of viral particles [5].

OBR-London brings together experts from industry and academia and a co-moderator from Nature to discuss the commercial potential of the latest developments in gene therapy and give candid opinions on the case to continue spending taxpayers’ and shareholders’ money on this research area.